[Novel therapies for acute myeloid leukemia based on genomic aberrations]

Rinsho Ketsueki. 2019;60(6):594-599. doi: 10.11406/rinketsu.60.594.
[Article in Japanese]


Standard treatment for acute myeloid leukemia (AML) comprises (1) induction therapy with both cytarabine and anthracycline and (2) consolidation therapy that is modified according to patients' conditions, including prognostic factors. However, this strategy is not satisfactory, especially for elderly patients. Novel technologies have revealed several driver mutations of numerous critical genes in AML, which can be targeted by novel drugs; the discovery of such targetable genes and the development of novel drugs have evolved the treatment strategy for AML. We should always monitor these advances in hematology. In the United States, the FDA has already approved several new drugs for AML, including FLT3 inhibitors and IDH neoenzyme inhibitors. In Japan, gilteritinib, an FLT3 inhibitor, was also approved at the end of 2018. These promising drugs will facilitate performing "precision medicine" on patients with AML soon.

Keywords: AML; FLT3; Genomic aberrations; IDH.

Publication types

  • Review

MeSH terms

  • Cytarabine / therapeutic use
  • Enzyme Inhibitors / therapeutic use*
  • Genomics
  • Humans
  • Japan
  • Leukemia, Myeloid, Acute / drug therapy*
  • Leukemia, Myeloid, Acute / genetics*
  • Protein Kinase Inhibitors / therapeutic use


  • Enzyme Inhibitors
  • Protein Kinase Inhibitors
  • Cytarabine